Roche's biotechnology subsidiary Genentech has reported positive interim results from the Phase III HAVEN 2 clinical trial of emicizumab for the treatment of children with haemophilia A and inhibitors to factor VIII.

Emicizumab is a bispecific monoclonal antibody being developed to combine factors IXa and X with proteins necessary for the stimulation of the natural coagulation cascade, as well as restore the blood clotting process.

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The results from the single-arm, multi-centre, open-label HAVEN 2 trial indicated that emicizumab prophylaxis has significantly decreased the number of bleeds over time after treatment for 12 weeks.

The Phase III trial will assess the efficacy, safety and pharmacokinetics of once-weekly emicizumab given subcutaneously to 19 children younger than 12 who need treatment with bypassing agents.

"A total of 60 children will be recruited by the time of final product analysis, following 52 weeks of treatment."

A total of 60 children will be recruited by the time of final product analysis, following 52 weeks of treatment.

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The trial's objectives include assessment of number of bleeds over time, safety, pharmacokinetics, health-related quality of life (HRQoL) and proxy HRQoL.

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The results from the HAVEN 2 trial were found to be consistent with the data from the Phase III HAVEN 1 trial conducted in 109 adults and adolescents aged 12 and above with inhibitors to factor VIII.

Emicizumab is being assessed further in Phase III HAVEN 3 and HAVEN 4 clinical trials.

The HAVEN 3 trial will evaluate once a week or once every other week emicizumab prophylaxis in haemophilia A patients aged 12 and above without inhibitors to factor VIII, while HAVEN 4 will include dosing every four weeks in patients 12 years and older with or without inhibitors to factor VIII.